Introduction

OBJECTIVES: To (a) enable decision making based on indicators of treatment outcomes for multiple myeloma (e.g., overall survival, progression-free survival, etc.) and markers of clinical efficacy (e.g., complete response, partial response, progressive disease, etc.); (b) coordinate oncology care and health plan medical and pharmacy management services to improve outcomes for patients with multiple myeloma; (c) enable the use of decision support tools to appropriately invest resources and reduce treatment variability with multiple myeloma therapies; (d) construct a benefit design model for multiple myeloma drugs; (e) recommend methods to improve patient outcomes with supportive care for multiple myeloma within a health plan setting; and (f) implement accurate and appropriate counsel, as part of the treatment team, that will improve patient adherence to treatment recommendations. SUMMARY: The first article in this supplement, “Identifying Indicators of Outcomes and Implementing Treatment Pathways,” reviews outcomes measures typically used in phase 3 clinical trials investigating novel oncology therapies and how these measures influence clinical decision making in the treatment of multiple myeloma. The second article, “Applying Oncology Formulary and Benefit Design Innovations to the Management of Multiple Myeloma in the Managed Care Setting,” discusses how comparative effectiveness research is used to generate data that can be utilized by policy makers, plan administrators, payers, and patients to identify therapies that provide the greatest value. The third article, “Multiple Myeloma: Supportive Care Requirements and Coordination of Patient-Centered Care,” identifies the elements of supportive care for multiple myeloma and discusses techniques to keep the patient experience as the focal point of the treatment plan. CONCLUSIONS: Multiple myeloma is representative of a disease in which the introduction of novel therapies has increased survival and patient quality of life. Increasing use of these innovative yet expensive drugs has motivated efforts to redesign the oncology pharmacy benefit in a way that promotes both enhanced clinical outcomes and cost control. This process relies on robust economic and clinical data; however, these data are limited. To determine the best value-based strategies for the treatment of patients with multiple myeloma, managed care decision makers must continue to assess the evolving landscape of treatment options and consider both clinical outcomes and treatment costs in their analyses.


Introduction
Jeffrey D. Dunn, PharmD, MBA M ultiple myeloma is a malignant neoplasm of plasma cells that accumulate in bone marrow, leading to bone destruction and marrow failure. More than 20,000 new cases are diagnosed annually in the United States, with an estimated 10,710 deaths resulting from the disease. 1 Although currently considered incurable, the treatment of multiple myeloma has improved dramatically over the past decade. The introduction of new treatment strategies (e.g., induction and maintenance), innovative new drugs (e.g., thalidomide, lenalidomide, bortezomib), new drug combination regimens (e.g., bortezomib/cyclophosphamide/dexamethasone, melphalan/prednisone/lenalidomide), novel interventions (e.g., autologous stem cell transplantation), and improved supportive care (e.g., bisphosphonates, pain management) has significantly expanded the therapeutic armamentarium. 2 It is estimated that survival now ranges from 1 year to more than 10 years, with a median survival of approximately 45 to 60 months. 1 With the availability of new agents and techniques, clinical decision making in multiple myeloma has become increasingly complex. Clinicians require data-ideally, data comparing survival outcomes of all treatment options with alternative interventions-to guide health care policy, benefit design, and therapeutic selection, but these data are rarely available. Consequently, comparative effectiveness research (CER) is frequently used to generate data that can be used to assess the value of competing therapies.
In an effort to improve the quality of patient care, health care decision making has become more evidence based and focused on outcomes. Ironically, in the headlong rush to improve the delivery of high-value care, there is a tendency to lose sight of the individual patient. Hence, several patient-centered research initiatives under way are designed to provide patients with the information they need to become fully informed consumers of health care products and services. 3 Oncology pharmacotherapy and benefit design is rapidly evolving, providing both challenges and opportunities for managed care stakeholders. Payers must quickly assess the value of new treatments and work with plan administrators to modify the oncology benefit in a way that provides access to the novel intervention while simultaneously controlling costs. While new treatment options often promise improved disease control and possibly increased survival, patients and their physicians must work together to find the most appropriate fit of the new agent into the therapeutic regimen. Using multiple myeloma as an example, this supplement was designed to review the ongoing changes in oncology pharmacotherapy and provide insights on how to manage the impact of these changes. The first article, "Identifying Indicators of Outcomes and Implementing Treatment Pathways," provides a brief overview of how innovations in drug therapy have impacted the treatment of patients ABSTRACT BACKGROUND: The treatment of multiple myeloma continues to evolve as innovative therapies increase survival times and patient quality of life. The increasing availability and utilization of these novel therapies challenge health plan administrators to create a pharmacy benefit structure that minimizes barriers to patient access while simultaneously managing costs.
OBJECTIVES: To (a) enable decision making based on indicators of treatment outcomes for multiple myeloma (e.g., overall survival, progressionfree survival, etc.) and markers of clinical efficacy (e.g., complete response, partial response, progressive disease, etc.); (b) coordinate oncology care and health plan medical and pharmacy management services to improve outcomes for patients with multiple myeloma; (c) enable the use of decision support tools to appropriately invest resources and reduce treatment variability with multiple myeloma therapies; (d) construct a benefit design model for multiple myeloma drugs; (e) recommend methods to improve patient outcomes with supportive care for multiple myeloma within a health plan setting; and (f) implement accurate and appropriate counsel, as part of the treatment team, that will improve patient adherence to treatment recommendations.
SUMMARY: The first article in this supplement, "Identifying Indicators of Outcomes and Implementing Treatment Pathways," reviews outcomes measures typically used in phase 3 clinical trials investigating novel oncology therapies and how these measures influence clinical decision making in the treatment of multiple myeloma. The second article, "Applying Oncology Formulary and Benefit Design Innovations to the Management of Multiple Myeloma in the Managed Care Setting," discusses how comparative effectiveness research is used to generate data that can be utilized by policy makers, plan administrators, payers, and patients to identify therapies that provide the greatest value. The third article, "Multiple Myeloma: Supportive Care Requirements and Coordination of Patient-Centered Care," identifies the elements of supportive care for multiple myeloma and discusses techniques to keep the patient experience as the focal point of the treatment plan.
CONCLUSION: Multiple myeloma is representative of a disease in which the introduction of novel therapies has increased survival and patient quality of life. Increasing use of these innovative yet expensive drugs has motivated efforts to redesign the oncology pharmacy benefit in a way that promotes both enhanced clinical outcomes and cost control. This process relies on robust economic and clinical data; however, these data are limited. To determine the best value-based strategies for the treatment of patients with multiple myeloma, managed care decision makers must continue to assess the evolving landscape of treatment options and consider both clinical outcomes and treatment costs in their analyses.

■■ Conclusion
Oncology pharmacy has been identified as a priority area for managed care for optimizing clinically and economically appropriate care for their members. With innovative treatments, many patients are living longer and thus require longterm therapy. With increasing duration of therapy, utilization and costs are increasing rapidly. 5 To more effectively manage this utilization and costs, as well as to ensure continued delivery of high-quality care, managed care organizations must take the lead in making oncology pharmacy treatment and benefit decisions that will result in optimal patient outcomes.